Mucopolysaccharidosis (MPS)
Conditions
Brief summary
Currently, treatments for mucopolysaccharidoses (MPS) are pharmacological and predominantly focus on the physical symptoms of the disease. In the general population and individuals with disabilities, increasing physical activity levels and reducing sedentary time can be an effective, non-pharmacological treatment to improve mental health and quality of life. Such interventions have not yet been developed or evaluated in people with MPS. The aim of this study is to co-design a physical activity and sedentary behaviour intervention tailored to the needs of adults with MPS. The study will seek to gain the expertise of adults with MPS, specialist stakeholders (physicians and clinical nurse specialists) and lay specialist stakeholders (family and friends of adults with MPS and members of staff and volunteers at the Society for Mucopolysaccharide Diseases). A range of views and experiences of physical activity and sedentary behaviour will be explored via focus groups (with individuals with MPS and lay specialist stakeholders) and semi-structured interviews (with specialist stakeholders). The information gathered from the focus groups and interviews will then be utilised to inform participatory workshops (with individuals with MPS) to test intervention concepts. Data from these activities will inform the design of a future intervention.
Interventions
Qualitative study (no intervention delivered)
Sponsors
Brunel University
University College London Hospitals
Northern Care Alliance NHS Foundation Trust
Royal Free Hospital NHS Foundation Trust
Study design
Observational model
OTHER
Time perspective
CROSS_SECTIONAL
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
Yes
Inclusion criteria
Adults with Mucopolysaccharidosis will be eligible to participate in the study if they meet the following inclusion criteria: * Diagnosed with any type of Mucopolysaccharidosis. * Aged ≥ 18 years old. * Lives in the United Kingdom. * Deemed safe to participate by a clinician (for participatory workshops).
Exclusion criteria
for all participants will be as follows: * Incapacity to provide informed consent. * A level of intellectual disability that would prevent the individual from making a meaningful contribution to the focus groups or workshops. * Unable to communicate in English to a sufficient level to permit engagement in the study. The circumstances in which an individual may be deemed ineligible to take part in the participatory workshops based on the clinician's assessment are as follows: * Severe joint pain. * Neurological compromise. * Stage 4 heart failure or other condition resulting in breathlessness to a level they cannot participate. * Other clinical condition which dominates the clinical picture to the extent that it dominates the symptoms and influences the findings (e.g. stroke). Specialist stakeholders will be members of staff at NHS specialist centres for Mucopolysaccharidosis who are directly involved in healthcare provision to the target population. Lay specialist stakeholders will be individuals who have been involved in supporting adults with Mucopolysaccharidosis, such as family, friends, and members of staff and volunteers at the Society for Mucopolysaccharide Diseases.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Focus groups (adults with Mucopolysaccharidosis) | 7 months | Themes describing adults with Mucopolysaccharidosis' views and experiences of physical activity and sedentary behaviour- Emergence of themes describing participants' views and experiences from analysis of focus groups (using the Framework Method). |
| Focus groups (lay specialist stakeholders) | 7 months | Themes describing lay specialist stakeholders' views and experiences of physical activity and sedentary behaviour- Emergence of themes describing participants' views and experiences from analysis of focus groups (using the Framework Method). |
| Semi-structured interviews (specialist stakeholders) | 7 months | Themes describing specialist stakeholders' views and experiences of physical activity and sedentary behaviour- Emergence of themes describing participants' views and experiences from analysis of interviews (using the Framework Method). |
| Participatory workshops (adults with Mucopolysaccharidosis) | 7 months | Themes describing adults with Mucopolysaccharidosis' views and experiences of initial intervention concepts- Emergence of themes describing participants' views and experiences from analysis of participatory workshops (using the Framework Method). |
Countries
United Kingdom
Contacts
CONTACTDaniel Bailey
CONTACTSarah Gosling
Outcome results
None listed